October 06, 2006: NIH Completes Collaborative Phase II Study with Santhera's compound SNT-MC17/idebenone in Friedreich's Ataxia (FRDA)
Bethesda, Maryland, USA, and Liestal, Switzerland - The US National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) and Santhera Pharmaceuticals, a Swiss speciality pharmaceutical company with a focus on neuromuscular diseases, announced today the completion of their six month double-blind placebo-controlled Phase II clinical trial with SNT-MC17/idebenone in Friedreich's Ataxia (FRDA). This trial was primarily designed to establish additional safety data in young patients with FRDA, particularly at higher doses, and to study the potential efficacy of SNT-MC17/idebenone on neurological symptoms and findings in this disease.
The trial enrolled a total of 48 patients, with 12 patients in each of three dose groups and a placebo group. Data from this trial support the favourable safety profile of the compound. Preliminary analysis of the trial results suggests that SNT-MC17/idebenone has a trend toward dose proportional improvement in neurological parameters in FRDA patients as determined by the International Cooperative Ataxia Rating Scale (ICARS), a semi-quantitative scale based upon 19 test parameters that assess sensory and motor skills.
These new data reinforce Santhera's belief that SNT-MC17/idebenone has the potential to provide a possible treatment for this devastating disease. The NINDS will present the data from this collaborative trial to the scientific, medical and patient community at the upcoming 3rd International Friedreich's Ataxia Scientific Conference, to be held in Bethesda, Maryland, from November 10 to 12, 2006.
Dr. Nicholas Di Prospero, principal investigator of the study, said: "In this trial we found SNT-MC17/idebenone to be safe and well tolerated and we are encouraged to pursue further trials to establish its positive effect on neurological function in young FRDA patients."
"We are pleased that Santhera has collaborated with us in this study and is committed to complete clinical development of SNT-MC17/idebenone for FRDA in the US. We are especially grateful to the patients and their families who participated in this important clinical study", commented Dr. Kenneth H. Fischbeck, Chief of the Neurogenetics Branch at NINDS.
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About Friedreich's Ataxia
Friedreich's Ataxia (FRDA) is a rare but severe genetic neuromuscular disorder that results in the degeneration of an individual's nerve and muscle tissue. This disorder causes loss of muscle control, uncoordinated movements, muscle wasting and thickening of heart walls which frequently lead to a shortened life span. FRDA affects both Caucasian males and females equally and it is estimated that about 20,000 patients suffer from the disease in both North America and Europe. Average life expectancy for FRDA patients is limited to approximately 35 to 50 years.
The disorder results from a genetic defect in the gene encoding for frataxin. Reduced levels of this protein ultimately result in impaired energy production in mitochondria, the cells' energy production centers, and elevated oxidative stress. Tissues that have the highest need for energy, in particular nerve and cardiac tissues, are primarily affected by frataxin deficiency resulting in pathological changes in heart muscle anatomy and function and loss of nerve cells. SNT-MC17/idebenone is believed to improve the balance and flow of electrons within the mitochondria, therefore increasing the energy production within nerve and muscle cells of FRDA patients, protecting these cells from cell death. A number of clinical trials provide strong evidence that SNT-MC17/idebenone may offer an effective treatment option for FRDA associated heart wall thickening (cardiomyopathy). In addition, data from this collaborative NIH Phase II clinical trial suggest positive effects on neurological function.
About US National Institute of Neurological Disorders and Stroke (NINDS)
The NINDS, a component of the National Institutes of Health in Bethesda, MD, is the nation's leading agency for research on the brain and nervous system. More information about the NINDS is available at its website, www.ninds.nih.gov.
The National Institutes of Health (NIH) - The Nation's Medical Research Agency - includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.
Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focusing on the discovery, development and marketing of small molecule pharmaceutical products for the treatment of severe neuromuscular diseases. Santhera's vision is to become a leading specialty pharmaceutical company offering therapies for a number of indications in this area of high unmet medical need which includes many orphan indications with no current therapy.
Santhera currently has four clinical-stage development programs, three of which are investigating its lead compound, SNT-MC17/idebenone, in the treatment of Friedreich's Ataxia (FRDA), Duchenne Muscular Dystrophy (DMD) and Leber's Hereditary Optic Neuropathy (LHON). The fourth clinical program is investigating JP-1730/fipamezole for the treatment of Dyskinesia in Parkinson's Disease (DPD) in cooperation with Juvantia. The most advanced program, SNT-MC17/idebenone in FRDA, has entered pivotal Phase III clinical development; the other clinical programs are in Phase II. Santhera's drug pipeline comprises another three preclinical programs in cancer cachexia, DMD and type 2 diabetes (out licensed to Biovitrum).
For further information on Santhera, please visit www.santhera.com.
For further information, contact:
US National Institute of Neurological Disorders and Stroke
Dr. Kenneth H. Fischbeck, Chief of the Neurogenetics Branch
Phone +1 301 435 9318
Dr. Nicholas Di Prospero, principal investigator of the study
Phone +1 301 435 9287
Thomas Meier, Chief Scientific Officer
Phone +41 61 906 89 87
Thomas Staffelbach, VP Public & Investor Relations
Phone +41 61 906 89 47
Media contact: Citigate
Phone +44 207 638 9571
Phone +44 207 638 9571